Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
GlaxoSmithKline
Age
9–any
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a sponsor approved DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by treatment with GSK2402968.
Age 9 years old or greater at Screening;
Male;
Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;
Life expectancy at least three years;
Willingness and ability to comply with all protocol requirements and procedures;
QTc \<450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period). Note: QTc may be either QTcB or QTcF, machine read or manual overread;
Subjects must be willing to use adequate contraception (condoms or abstinence), from Screening until at least 5 months after the last dose of study drug;
Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).
You may not be if
Any additional mutation (such as an additional missing exon for DMD) that cannot be treated with GSK2402968;
Current or history of liver or renal disease;
Acute illness within 4 weeks of anticipated administration of study medication, which may interfere with study assessments;
Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, idebenone or other forms of Coenzyme Q10, within 6 months of the first administration of study medication;
Start of glucocorticosteroids within 6 months or non-stable use of glucocorticosteroids within 3 months of the anticipated first administration of study medication;
Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at Screening;
Symptomatic cardiomyopathy;
Use of alcohol from Screening through to the 1 month Follow-up visit ;
